Mission Statement

As part of the federal government’s National Institutes of Health (NIH), the National Eye Institute’s mission is to “conduct and support research, training, health information dissemination, and other programs with respect to blinding eye diseases, visual disorders, mechanisms of visual function, preservation of sight, and the special health problems and requirements of the blind.”

Leber Congenital Amaurosis (LCA)

When people lose the ability to see, how do the visual parts of the brain change in response? And if they regain their sight, are the changes reversed? Researchers at the University of Pennsylvania took a rare opportunity to address these...

Gene therapy for Leber congenital amaurosis (LCA), an inherited disorder that causes vision loss starting in childhood, improved patients’ eyesight and the sensitivity of the retina within weeks of treatment. Both of these benefits, however,...

Three young adults with an inherited form of blindness showed evidence of improved day and night vision following a specialized gene transfer procedure in a phase 1 clinical trial funded by the National Eye Institute (NEI), part of the National...

Three young adults with Leber Congenital Amaurosis-a severe degenerative disease of the retina caused by a mutation in the RPE65 gene-reported improvements in vision after undergoing a specialized gene transfer procedure. In October and December...

NEI support leading to gene therapy clinical trials for people with Leber congenital amaurosis (LCA) consisted of significant efforts by investigators in the intramural laboratories at the NEI as well as many extramural investigators at...

Two groups of investigators have reported independently on their initial observations from Phase I clinical trials of gene transfer for Leber congenital amaurosis (LCA) caused by mutations in the RPE65 gene. The two papers appear in the April 27...

The National Eye Institute (NEI) of the National Institutes of Health (NIH) is supporting a phase I clinical trial to assess the safety of gene transfer in treating people with a form of Leber congenital amaurosis (LCA). People with the LCA...